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Phase I/II Study of Induction of Stable Mixed Chimerism After Bone Marrow Transplantation From HLA-Identical Donors in Children With Sickle Cell Disease


Phase 1/Phase 2
N/A
16 Years
Not Enrolling
Both
Sickle Cell Anemia

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Trial Information

Phase I/II Study of Induction of Stable Mixed Chimerism After Bone Marrow Transplantation From HLA-Identical Donors in Children With Sickle Cell Disease


PROTOCOL OUTLINE: This is a multicenter study. Patients undergo total body irradiation on
day 0, followed by allogeneic bone marrow transfusion. Patients also receive fludarabine IV
daily and cyclosporine IV twice a day on days -1 to 1. Patients then receive oral
cyclosporine on days 1-90, and oral mycophenolate mofetil twice a day on days 0-27.

Patients are followed for 100 days, monthly for 6 months and then annually for 2 years.

Inclusion Criteria


PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Diagnosis of sickle cell anemia with clinically severe disease manifestations defined by:
Recurrent painful events (at least 2 painful events in past year) which cannot be
explained by other causes Pain lasts at least 4 hours Requires treatment with parenteral
narcotics, equianalgesic dose of oral narcotics, or parenteral nonsteroidal
antiinflammatory drugs Acute chest syndrome (ACS) with at least 2 episodes within past 2
years that required hospitalization, oxygen, and RBC transfusion Any combination of
painful events and ACS episodes that total 2 events within the past year Abnormal cerebral
MRI, abnormal angiography (MR or conventional), and abnormal neuropsychologic testing
performance

No stage III or IV sickle cell lung disease

Genotypically HLA identical sibling donor available

--Prior/Concurrent Therapy--

No prior transfusions with greater than 5 units RBC

--Patient Characteristics--

Performance status: Karnofsky 70-100%

Hepatic:

- No active hepatitis

- No moderate/severe portal fibrosis

Renal: Glomerular filtration rate at least 30% predicted for age

Neurologic:

- No severe residual functional neurologic impairment

- Hemiplegia alone allowed

Other:

- HIV negative

- Not pregnant or nursing

- Fertile patients must use effective contraception

Type of Study:

Interventional

Study Design:

Primary Purpose: Treatment

Principal Investigator

Mark Walters

Investigator Role:

Study Chair

Investigator Affiliation:

Children's Hospital of Oakland

Authority:

Unspecified

Study ID:

199/14243

NCT ID:

NCT00004485

Start Date:

December 1999

Completion Date:

Related Keywords:

  • Sickle Cell Anemia
  • genetic diseases and dysmorphic syndromes
  • hematologic disorders
  • rare disease
  • sickle cell anemia
  • Anemia
  • Anemia, Sickle Cell

Name

Location

Fred Hutchinson Cancer Research CenterSeattle, Washington  98109
Children's Hospital of OaklandOakland, California  94609-1809